Late-breaking science exploring breakthrough strategies in the heart failure (HF) journey were the focus of a special session Nov. 5 during AHA 2022 in Chicago. Researchers presented findings from the CRISPR, COACH, PRO-HF and IRONMAN trials.

In CRISPR, a single intravenous infusion of NTLA-2001, a novel gene-editing therapy based on CRISPR/Cas9, significantly reduced abnormal levels of the transthyretin protein (TTR) by more than 90% in patients with ATTR amyloid cardiomyopathy after 28 days. The therapy was generally well-tolerated and the majority of adverse events were mild, according to researchers. "This is the first-ever human trial of gene editing in vivo, or in the body, and our study proves that gene editing in the human body is possible and also safe in the short term," said Julian D. Gillmore, MD, PhD, who presented the findings. "These results indicate that intravenous (IV) NTLA-2001 is a potential new treatment option that may stop disease progression in patients with ATTR amyloid cardiomyopathy or even bring about improvement. However, further research is needed to establish long-term safety of NTLA-2001 and to continue to monitor and evaluate the potential effects of markedly reduced TTR levels on patients' clinical outcomes."

Results from COACH, which were also simultaneously published in the New England Journal of Medicine, found that the systematic use of a hospital-based point-of-care tool to support clinical decision-making, followed by rapid follow-up in an outpatient clinic, led to a lower risk of death or hospitalization for cardiovascular causes within 30 days among patients with acute HF seeking emergency care. Compared with usual care, "implementation of this approach across health systems may provide a pathway for early and safe discharge from the hospital or emergency department and improved patient outcomes," said Douglas S. Lee, MD, et al.

In PRO-HF, which was simultaneously published in Circulation: Heart Failure, researchers observed that routine collection of patient-reported health status using the Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12) in the HF clinic led to improved accuracy of clinician assessments of patients' health status. Correspondingly, researchers also noted that patients believed their clinicians better understood their symptoms. "The downstream impact of patient-reported health status assessment on clinical decision-making and outcomes remains to be seen and should be further investigated," said Alexander T. Sandhu, MD, et al.

Meanwhile, IRONMAN results, also simultaneously published in The Lancet, found that repeated IV administration of iron reduced the risk of hospitalization for HF and cardiovascular death by approximately 20% in people with HF and iron deficiency. Researchers said treatment with IV iron appeared to be safe and well-tolerated, and people who received it reported improved well-being based on quality-of-life questionnaires four months after treatment. "This study builds on existing evidence such that intravenous iron may benefit a broad range of people with HF, including those who are hospitalized, recently discharged or attending office or out-patient clinic appointments," said Paul Kalra, MD, who presented the findings.

Clinical Topics: Cardiovascular Care Team, Heart Failure and Cardiomyopathies, Acute Heart Failure, Heart Failure and Cardiac Biomarkers

Keywords: AHA Annual Scientific Sessions, AHA22, Amyloid Neuropathies, Familial, Cardiomyopathies, CRISPR-Cas Systems, Ferric Compounds, Gene Editing, Health Services Accessibility, Heart Failure, Hospitalization, Natriuretic Peptide, Brain, Patient Reported Outcome Measures, Stroke Volume

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